In this section, we look beyond treatment to explore new and interesting topics related to managing MM through the lens of RWE. From clinical trial eligibility to the impact of the COVID-19 pandemic on MM management, you’ll find a diverse range of content. The implementation of RWE to better understand the MM treatment landscape and MM management is a fast-moving field. This page will be periodically updated as more information becomes available, so please check back here to review updated information.
MANAGING PATIENTS WITH MULTIPLE MYELOMA BEYOND THE CLINICAL TRIAL SETTING: REAL-WORLD TREATMENT CHOICES
Which factors are important to patients in the RW setting?
“Efficacy does not necessarily mean the same thing to different patients.” 1
Each patient’s experience of MM is unique; therefore ‘efficacy’ may have a different meaning to different patients. The traditional definition of efficacy does not encompass many facets of the treatment experience that can impact the effectiveness which can be achieved. Safety, tolerability, feasibility, QoL and treatment satisfaction all have an impact on the outcomes that are obtainable with any given treatment regimen. To derive full benefit from a treatment, several factors that MM patients associate with the effectiveness of a treatment in a RW setting should be considered. These include symptom burden, side effects, daily activities, financial toxicity, treatment convenience/route of administration, and patient preferences. A holistic assessment should be considered to appropriately define efficacy and guide treatment for each patient, gathering input from the patient, their family, the clinical trial team, and other support teams.
Important treatment factors for RW MM patients1
Adapted from Terpos E, at al. 2021.1
Improving the link between clinical trial efficacy and real-world effectiveness:2
- Clinical trials could have broader inclusion criteria and RW studies should be defined to include data sources, minimal data requirements, and a variety of contributing centers
- Parallel collection of both clinical trial and RW data
- Standardized core set of outcomes, including more PRO and QoL data
- Incorporate assessment of PRO and QoL data routinely to allow for quicker access to drugs
- The value of PRO should be cascaded from specialized centers
- Consider parallel post-marketing trials to gather additional data to augment the clinical dataset initially available for regulatory submissions
Safety, tolerability, feasibility, QoL, treatment satisfaction, and efficacy, could be used in combination to determine treatment effectiveness. Such an approach may help ensure that patients with MM in the RW are represented in clinical trials so that they can access better treatments, which may ultimately improve patient QoL.
1. Terpos E et al. Blood Cancer Journal. 2021;11:40. 2. FDA. Framework for FDA’s Real-world evidence program. Available at: https://www.fda.gov/media/120060/download. Last accessed August 2021.
MM, multiple myeloma; PRO, patient-reported outcome; QoL, quality of life; RW, real-world.